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Mesalamine for Colorectal Cancer Prevention Program in Lynch syndrome

Primairy Objective* To test whether 5-ASA reduces the occurrence of colonic benign or malignant neoplasia compared to placebo in Lynch syndrome (LS) patients as detected by any colonoscopy until the end of study.Secondary Objectives* To test wheter…

A Phase 3 Efficacy and Safety Study of Ataluren
(PTC124 ®) in Patients with Nonsense Mutation Cystic
Fibrosis

To evaluate the ability of ataluren to improve pulmonary function relative to placeboTo determine the effect of ataluren on:1. Pulmonary symptoms2. General well-being3. Health-related Quality Life (HRQL)

A randomized, double-blind, placebo controlled study of canakinumab in patients with Hereditary Periodic Fevers (TRAPS, HIDS, or crFMF), with subsequent randomized withdrawal/ dosing frequency reduction and open-label long term treatment epochs

Primary: The primary objective of the randomized treatment epoch and for the overall study is to demonstrate that subcutaneous canakinumab administered every 4 weeks is superior to placebo in achieving a clinically meaningful reduction of disease…

A three-arm, randomized, double-blind, placebo-controlled study of the efficacy and safety of two trough-ranges of everolimus as adjunctive therapy in patients with tuberous sclerosis complex (TSC) who have refractory partial-onset seizures

Primary: To compare the reduction in frequency of partial-onset seizures on each of two trough ranges of everolimus (3-7 ng/mL and 9-15 ng/mL) versus placebo in patients with TSC who are taking one to three AEDs. Key secondary: Ability to completely…

A Randomized, Placebo- and Active-Controlled, Double-Blind, 4-way Crossover Design, Thorough ECG Study of VX-770 in Healthy Adult Subjects

Part A:To evaluate the safety and tolerability of increasing doses of VX-770 up to 450 mg (q12h) in healthy male subjects.Part B:To determine if therapeutic or supratherapeutic systemic exposure to multiple doses of VX 770 prolongs the mean…

A 2-part, randomized, double-blind, placebo-controlled, sequential group, dose-escalation study to assess the safety, tolerability and pharmacokinetics of single and multiple ascending oral doses of GLPG2737 in healthy male sub.

The purpose of the study is to investigate how safe GLPG2737 is and how well GLPG2737 is tolerated. It will also be investigated how quickly and to what extent GLPG2737 is absorbed into, distributed in, and eliminated from the body (this is called…

Multi-center, randomized, double-blind, placebo-controlled phase 2 study to assess the safety, tolerability and early signs of efficacy of tid orally administered BAY63-2521 in adult deltaF508
homozygous Cystic Fibrosis patients

The aim of this trial is to test in a first clinical study the above mentioned hypothesis that BAY63-2521 can at least partially correct deltaF508-CFTR function. Therefore, we plan to include patients with Cystic Fibrosis, who are homozygous for the…

A randomized, double-blind, placebo-controlled, multiple ascending dose study to examine the safety, tolerability and pharmacokinetics of orally administered PHA-022121 in healthy subjects.

Primary ObjectiveTo assess the tolerability and safety of multiple ascending oral doses of PHA-022121 administered after a standard caloric meal in healthy adult subjects.To assess the PK characteristics of PHA-022121 after administration of…

A randomized, double-blind, placebo-controlled, single ascending dose study to examine the safety, tolerability and pharmacokinetics of orally administered PHA-022121 in healthy subjects.

Primary ObjectiveTo assess the tolerability and safety of single ascending oral doses of PHA-022121 administered after a standard meal and of a single 40 mg dose under fasted conditions in healthy adult subjects.To assess the PK characteristics of…

A multicenter, double-blind, randomized, placebo-controlled, parallel-arm study to investigate the efficacy and safety of subcutaneous administration of CSL312 (garadacimab) in the prophylactic treatment of hereditary angioedema

The primary objective of this study is to evaluate the efficacy of SC administration of CSL312 as prophylaxis to prevent HAE attacks in subjects with HAE.The secondary objectives of the study are:1. To characterize the clinical efficacy of SC CSL312…

Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2002 in Adults with Hereditary Angioedema (HAE)

This study has been transitioned to CTIS with ID 2024-512317-40-00 check the CTIS register for the current data. Phase I Primary Objective• To evaluate the safety of NTLA-2002 and identify dose(s) for use in Phase 2Secondary Objectives• To evaluate…

A phase 1, randomized, double-blind, placebo-controlled, safety, tolerability, pharmacokinetic and biomarker study of multiple ascending doses of ESB1609 in healthy volunteers.

To assess the safety and tolerability profile of escalating dose levels of ESB1609 when administered as once daily (qd) oral doses for up to 25 days in healthy volunteers.

A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients with Glycogen Storage Disease Type Ia

This study has been transitioned to CTIS with ID 2023-508750-25-00 check the CTIS register for the current data. Primary: To evaluate the efficacy of DTX401 to reduce or eliminate dependence on exogenous glucose replacement therapy needed to…

Phase 1 Dose Escalation Study Assessing the Safety and Pharmacokinetics of PTC518 in Healthy Subjects

Primary ObjectivesPart 1:To characterize the safety and tolerability of single ascending doses of PTC518 in healthy subjects.Part 2:To characterize the safety and tolerability of PTC518 administered for 14 or up to 21 days in healthy subjects.Part 3…

A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-003 Administered Intrathecally in Patients With Huntington*s Disease

Primary objective:x To evaluate the safety and tolerability of WVE-003 in patients with Huntington's disease (HD)Secondary objectives:x To characterize the pharmacokinetics (PK) of WVE-003 in plasma x To characterize the concentration of WVE-…

A multi-centre, randomised, double-blind, placebo-controlled, Phase 2 study to investigate efficacy, safety and tolerability of SLN360 in participants with elevated lipoprotein(a) at high risk of atherosclerotic cardiovascular disease events.

The primary objective is to evaluate the effect of SLN360 on circulating levels of Lp(a) in participants with elevated Lp(a) at high risk of ASCVD events.The secondary objectives are to:• Evaluate safety and tolerability of SLN360 in participants…

Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients with Alexander Disease

This study has been transitioned to CTIS with ID 2024-510603-11-00 check the CTIS register for the current data. Primary Objective: To evaluate the efficacy of ION373 in improving or stabilizing gross motor function in patients with Alexander…

A multicenter, double-blind, randomized, placebo-controlled, parallel-group study to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.

Primary objectiveThe primary objective of the study is to determine the effect of lucerastat on neuropathic pain in subjects with Fabry disease (FD).Secondary objectives* To determine the effects of lucerastat on gastro-intestinal (GI) symptoms (…

A Phase IIb double-blind, randomised, placebo-controlled, multi-centre, confirmative three-way cross-over study on cognitive function with two doses of KH176 in subjects with a genetically confirmed mitochondrial DNA tRNALeu(UUR) m.3243A>G mutation.

To evaluate the effect of KH176 during a 4 week treatment period on the attention domain score of cognitive functioning, as assessed by the visual identification test of the Cogstate computerised cognitive testing battery.

A randomized placebo controlled, double-blind phase II study to explore the safety, efficacy and pharmacokinetics of sonlicromanol in children with genetically confirmed mitochondrial disease.

Primary Objective:To evaluate the effect of sonlicromanol on motor symptom severity in children with genetically confirmed mitochondrial disease affecting oxidative phosphorylation during a 6 month treatment period (GMFM).